Duchenne UK is the biggest funder of DMD research in the UK in the last three years. We are lean, effective and highly ambitious, totally focused on funding and accelerating research into treatments and a cure for Duchenne muscular dystrophy.

Who we are 

We are run by parents, fighting every day to find and speed up treatments for all our children and those living with DMD. Our approach is 100% focused on advancing research – and getting treatments into the clinic and to patients as quickly as possible. We actively seek out projects and possible therapies that could benefit this generation of patients. We invest globally in basic science and translational research. In the last 5 years we have raised over £10 million. We have travelled the world, meeting scientists, attending conferences, advocating on behalf not just of our sons but of the entire Duchenne commmunity, to advance and accelerate research.

What your money has helped us to fund 

We've spent £6.5m to date and committed a further £3.5m to accelerating research. We have achieved some incredible results, thanks to your support. We've co-funded two successful Phase I studies. We've funded pre-clinical work for a Gene Therapy trial. Find out more about these projects here. We're co-funding one doctor who is now running sixteen trials in the UK. We're also co-funding 16 clinical trial posts.

The 'multiplier effect' 

Duchenne UK has a pioneering and ambitious research approach, which means that the money you raise for us can go further.  On certain projects, we create a 'multiplier effect'.  Duchenne UK provides research spend to prove that a drug is ready for trial, which is then a catalyst for additional investment. What we're doing, is bridging the notorious valley of death in drug development, bringing promising therapies out of the laboratory and into the clinic where they can be tested on patients. 

Along with the Duchenne Research Fund, we invested $5million in a gene therapy project for Solid GT. That seed funding enabled them to raise $42m of private investment to fully fund further clinical trials.  Just three years later, that compound is NOW IN THE CLINIC being tested on boys with Duchenne!

We invested $1million of seed funding to co-fund the Phase I study of Vamorolone (VBP15). As a result of our investment, the company was able to raise a further $12million for the Phase II clinical trial which is NOW UNDERWAY!

Our Impact

We are very proud of the achievements of our collaborators. Please take a look here to see how our money has funded and accelerated research.